Are Some Diseases Better Suited for Stem Cell Therapy?
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Stem cell therapy holds significant promise for treating a range of diseases, particularly neurological and cardiovascular disorders. While SCT has shown potential in preliminary studies and small-scale clinical trials, further research is needed to address challenges such as immune rejection, ethical concerns, and long-term safety. The future of SCT looks promising, with ongoing advancements likely to enhance its efficacy and applicability across various medical conditions.
Stem cell therapy (SCT) is a promising field in regenerative medicine, aiming to treat various intractable diseases by regenerating or repairing damaged tissues. This synthesis explores the suitability of SCT for different diseases, focusing on neurological disorders, cardiovascular diseases, and other conditions.
Key Insights
- Neurological Disorders:
- SCT shows potential in treating neurodegenerative diseases like Parkinson’s, Alzheimer’s, multiple sclerosis, and stroke by replacing lost neurons and glial cells, and through neurotrophic factor release and immunomodulation2 3 4 6 7 10.
- Neural stem cell transplantation can enhance neuroplasticity, reduce neuroinflammation, and produce neurotrophic factors, making it a promising approach for neurodegenerative diseases2 4 10.
- Cardiovascular Diseases:
- SCT, particularly using human embryonic stem cells, shows promise in treating cardiovascular diseases such as congestive heart failure and congenital heart disease by differentiating into cardiomyocytes and vascular progenitors5 6.
- Stem cell delivery to the heart, as an adjunct to surgical treatments, may improve cardiac function and quality of life in congenital heart disease patients5.
- Diabetes Mellitus:
- Preliminary studies suggest that SCT could potentially treat diabetes by regenerating insulin-producing cells, although more research is needed to confirm efficacy and safety1.
- Retinal Degenerative Disorders:
- SCT has shown potential in treating retinal degenerative disorders by regenerating retinal cells, although clinical translation faces challenges such as immune rejection and genetic instability1.
- General Therapeutic Potential:
- Multipotent stem cells can treat various diseases due to their ability to self-renew and differentiate into multiple cell types, contributing to anti-inflammatory and anti-apoptotic effects8.
- Despite the potential, SCT faces challenges like ethical concerns, tumorigenicity, and the need for further research to understand the mechanisms and long-term effects1 9.
Are some diseases better suited for stem cell therapy?
Philippa Harding has answered Near Certain
An expert from University College London in Cell Biology, Developmental Biology, Stem Cells
Yes –Some types of stem cells produce new cells throughout life, so transplanting new stem cells is more effective at treating disease. Also, some areas of the body are more accessible, so easier to replace with new stem cells which integrate into the body to treat disease. For example, bone marrow transplants have been relatively successful, because blood (haematopoietic) stem cells continually produce new blood cells, and bone marrow can be extracted from donors and transplanted into patients with existing technologies. However, some parts of the body grow when you are young and do not repair/regenerate throughout adult life, so are more difficult to replace by transplanting stem cells into the body. One alternative way to treat some of these diseases is to grow stem cells into other cell types in the lab, and transplant the specialised cell type into the body, as has been done for certain cell types in the back of the eye (RPE) to treat age-related macular degeneration.
Are some diseases better suited for stem cell therapy?
Abigail Isaacson has answered Near Certain
An expert from King’s College London in Developmental Biology
Due to their ability to become specialised as numerous cell types, stem cells have primarily been studied in the context of tissue regeneration and replacement (See recent review: Stem Cells in the Treatment of Disease (nejm.org)). This encompasses the stimulation of a person’s own stem cells to promote the healing of native tissue as well as cell transplantation for the treatment of diseases characterised by the loss of cells, such as type 1 diabetes. The latter typically involves following carefully developed protocols for differentiating stem cells in vitro into multiple cell types, such as insulin-producing cells. Treatment of autoimmune diseases is another promising area of stem cell research, in which a class of stem cells (mesenchymal) has been shown to possess immunoregulatory effects on other cells. Most recently, the capability of stem cells to reduce inflammation has been harnessed to treat Covid-19-induced pneumonia, where its anti-inflammatory effects can weaken the associated “cytokine storm” (Transplantation of ACE2- Mesenchymal Stem Cells Improves the Outcome of Patients with COVID-19 Pneumonia (nih.gov)). Stem cells have also been used in conjunction with gene therapy to treat diseases such as HIV, where the overarching strategy is to generate genetically engineered cell populations that are resistant to HIV infection (Stem cell-based therapies for HIV/AIDS – ScienceDirect). Comparable methods have been used to target tumour cells using stem cell-derived immune cells expressing tumour-suppressing genes, capable of evading the host immune system (Frontiers | Mesenchymal Stromal/Stem Cells: A New Era in the Cell-Based Targeted Gene Therapy of Cancer | Immunology (frontiersin.org)).
Are some diseases better suited for stem cell therapy?
Vik Reebye has answered Likely
An expert from Imperial College London in Oncology
Mostly patients with degenerative diseases benefit the most from stem cell therapy. Degenerative is the key word here implying that the lack of a protein or enzyme being the cause of the disease. In these patients a marginal increase or replacement of that lost protein can drastically delay progression of the disease or improve their quality of life. In most circumstances, stem cell therapy will not be the only treatment given to a patient. There are areas of stem cell therapy where its benefits are yet to be fully explored. Patients who have had major knee or hip surgery are often directed towards stem cell therapy as an aid to accelerate bone healing.
Are some diseases better suited for stem cell therapy?
Barney Bryson has answered Near Certain
An expert from University College London in Neuroscience
Absolutely. In most cases, the goal of stem cell therapies is to replace a specific population of cells that have been lost or damaged due to disease or injury, such as liver cells (hepatocytes) in hepatitis patients and insulin secreting (Islet) cells in patients with type I diabetes. In order to do this, however, it is often necessary to be able to fully recapitulate the molecular programmes that are required to convert a stem cell into the desired specialized cell type to provide a source of replacement cells (note: a major exception is the transplantation of bone marrow stem cells, which are expanded and purified, before being used in their undifferentiated form). It is now possible to artificially transform stem cells into many, but not all, specialized cell types, however, in many cases it remains to be determined whether stem cell derived cells function in exactly the same way as the cells they are intended to replace and, importantly, whether they can successfully integrate into damaged tissue to effect a beneficial repair. Indeed, the more complex the tissue, the harder it will be to repair using stem cell therapies. For example, the ability of stem cell derived cardiomyocytes (heart muscle) cells to repair damage caused by heart attack was initially tested by delivery of dissociated cells, with limited success, but more recently, grafts of lab-grown, bioengineered scaffolds containing cardiomyocytes have begun to show more promise. A final thought is that scientists have figured out how to transform stem cells into many of the different neuronal cell types found in the brain (and are affected by specific diseases, such as motor neurons that are affected by amyotrophic lateral sclerosis, ALS), however, the extremely complex cytoarchitecture of the nervous system makes it one of the most difficult areas to target with stem cell therapies.
Are some diseases better suited for stem cell therapy?
Michael Telias has answered Near Certain
An expert from University of California, Berkeley in Neuroscience, Stem Cells, Vision Sciences
Stem cells can be used to replace or to regenerate.
In bone-marrow transplantation, we replace the abnormal stem cells in the bone marrow of the patient with new, fresh and healthy stem cells from a donor, reconstructing the blood system, free of cancer or other diseases.
In the future, we will be able to regenerate tissue that has been damaged using stem cells, such as skin burns, or neurons in neurodegenerative diseases like Parkinson’s or Alzheimer’s.
Are some diseases better suited for stem cell therapy?
Carolyn Carr has answered Near Certain
An expert from Oxford University in Heart Disease, Stem Cells
Stem cells act in two key ways. In ideal situations, stem cells can replaced the damaged cells in particular parts of the body. Stem cells can also release chemicals which can reduce inflammation and induce new blood vessel formation. Leukemia has been treated by stem cell therapy for many years, as stem cells are transplanted into the bone marrow, and stem cells have been injected into the knee of patients with osteoarthritis and shown beneficial results. Pancreatic beta-cells which make insulin can be formed from stem cells in the lab and it is hoped will be able to be used in the treatment of type 1 diabetes by transplanting new beta cells in diabetic patients. Making new heart cells in the lab is possible, but because the heart is a complex organ with multiple cell types which have to be arranged in line with contracting heart muscle, it is a more difficult challenge to introduce these new cells into the region of the heart where they are needed. However, injecting stem cells into the heart has been shown to be beneficial by damping down the inflammatory reaction and inducing the formation of new blood vessels. Other diseases are multi-factorial or affect the whole body and therefore are not suitable for stem cell therapy.
Are some diseases better suited for stem cell therapy?
Karl Willert has answered Likely
An expert from University of California, San Diego in Stem Cells, Developmental Biology, Cell Biology
Yes, I would say any condition where tissues, organs or body parts are damaged, diseased or dead will potentially benefit from stem cell treatments.
Modern medicine provides few treatments that reverse such conditions. I should add that stem cells hold promise not only for cell replacement therapies, e.g. fixing an infarcted heart, replacing insulin producing beta cells, repairing a damaged spine, or restoring vision. Stem cells also provide a cell-based system to study human diseases.
Currently, the best we can do is model human diseases in animals, such as mice, but such models are largely insufficient in recapitulating the disease. Stem cells can also be used to evaluate drug safety and efficacy. By generating specific cell populations from stem cells in a dish, we can examine to what extent a new drug impacts the behavior of these cells. For example, stem cells can be instructed to become heart cells that contract: if a particular drug interferes with this cellular behavior may be an indication that this drug will have adverse side effects on the heart.
Aside from providing a cell-based system for disease modeling and for drug testing/development, stem cells also offer a unique opportunity to scientists like me to study the early processes of human development and answer questions of how an undifferentiated stem cell acquires specialized functions.
Are some diseases better suited for stem cell therapy?
Christian Drapeau has answered Near Certain
An expert from Kalyagen in Stem Cells
Yes. Some organs and tissues repair more easily than others, in part due to the nature of the tissue and in part due to the extent of fine blood vasculature delivering stem cells to some tissues. For example, the liver, skin, and even the pancreas and the lungs can repair more easily than the heart, retina, or brain. But studies have clearly shown that stem cells play a crucial in the repair and renewal of all tissues. For example, increasing the number of circulating stem cells by stimulating endogenous bone marrow stem cell mobilization (ESCM) has been documented to support the repair of the pancreas to the point of improving diabetes (https://pubmed.ncbi.nlm.nih.gov/12639990/, https://pubmed.ncbi.nlm.nih.gov/17255204/, https://www.researchgate.net/publication/224829508). Similar results have been documented with Parkinson (https://pubmed.ncbi.nlm.nih.gov/24347771/), spinal cord injury (https://pubmed.ncbi.nlm.nih.gov/20202082/), and cardiomyopathy (https://pubmed.ncbi.nlm.nih.gov/11504914/, https://pubmed.ncbi.nlm.nih.gov/20670846/, https://pubmed.ncbi.nlm.nih.gov/15639484/), but ESCM has shown little results with macular degeneration, for example.
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